Updates

August 13, 2018August 13, 2018
FDA Grants Fast Track Status to Castle Creek’s CCP-020 for Treatment of EBS

Castle Creek Pharmaceuticals‘ investigational treatment CCP-020 (diacerein 1% ointment) was granted Fast Track designation by the U.S. Food and Drug Administration to treat a type ... Read more
August 8, 2018August 8, 2018
Joint Venture Plans to Advance Potential EB Therapy RGN-137 into Phase 3 Trial

RGN-137, an experimental therapy for wound healing in epidermolysis bullosa (EB), is expected to advance into a Phase 3 trial, backed by a joint ... Read more
August 6, 2018August 6, 2018
Amryt’s AP101 Granted Rare Pediatric Disease Designation for EB

Amryt Pharma’s investigational therapy AP101, designed to reduce the time it takes for skin wounds to close, was granted Rare Pediatric Disease designation by ... Read more
August 1, 2018August 1, 2018
Blocking Inflammation-linked PI3K-Delta Molecule Could Lead to EBA Therapy, Mouse Study Suggests

Treatment targeting a specific molecule involved in inflammation — called PI3K-delta — eased skin lesions and prevented blistering in mouse models of epidermolysis bullosa ... Read more
July 30, 2018July 30, 2018
Antibiotic Gentamicin May Benefit About 80% of Herlitz JEB Patients, Study Says

The commonly used antibiotic gentamicin (brand name Garamycin) can reverse the effects of about 80% of genetic mutations affecting the LAMB3 gene that cause Herlitz junctional epidermolysis ... Read more

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News

Cord Blood Platelet Gel Holds Potential to Treat Epidermolysis Bullosa, Study Shows
July 25, 2018July 25, 2018
A gel made from platelets — tiny blood cells involved in blood clotting — ...
News

Debra’s Family-oriented EB Care Conference Kicks Off Sunday in Arizona
July 20, 2018July 20, 2018
The 2018 Debra Care Conference — a U.S. gathering of epidermolysis bullosa (EB) patients, families, ...
News

Higher Levels of Granzyme B Play Key Role in EBA, Other Skin Blistering Diseases, Study Suggests
July 16, 2018July 16, 2018
An enzyme called granzyme B (GzmB) may be a key mediator of blistering in ...
News

Case Report Highlights Rare EBA Diagnosis in 21-Year-Old Patient
July 11, 2018July 11, 2018
A case report shows that epidermolysis bullosa acquisita (EBA), a rare form of epidermolysis ...
News

Phase 1/2 Trial of DEB Therapy Candidate QR-313 Now Enrolling Patients, ProQR Announces
July 9, 2018July 9, 2018
A Phase 1/2 clinical trial assessing investigational QR-313 as a treatment for dystrophic epidermolysis bullosa (DEB) has ...
News

Castle Creek Enrolls First Patient in Phase 1 Trial for CCP-020 as Treatment for EBS
June 27, 2018June 27, 2018
Castle Creek Pharmaceuticals recently enrolled its first patient in a Phase 1 clinical trial ...

2 New Mutations Found in Family with Epidermolysis Bullosa Pruriginosa, Case Study Reports
June 25, 2018June 25, 2018
Two new potentially damaging mutations were identified in a family with epidermolysis bullosa pruriginosa ...
Allogeneic Fibroblast Injections into Skin Lesions Are Safe, Effective for RDEB, Study Shows
June 18, 2018June 18, 2018
Allogeneic fibroblast injections are an effective and safe treatment strategy for non-healing wounds in ...
ProQR to Receive Up to $5 Million from Nonprofits to Develop QR-313 for DEB
June 13, 2018June 13, 2018
Serlopitant’s Potential to Manage Itch in EB Patients Shown in Exploratory Trial
June 6, 2018June 8, 2018
Abeona Opens Manufacturing Plant to Support EB-101, and Other Gene and Cell Therapy Candidates
June 4, 2018June 4, 2018
US ‘Right to Try’ Law Meets with Mix of Praise and Criticism, Including Among Those with Rare Diseases
June 4, 2018June 4, 2018

EB Treatment Candidate BPM 31510 Granted Orphan Drug Designation in US
May 30, 2018May 30, 2018
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to BERG’s ...
#ECRD2018 – Genome Editing Might Be ‘Cure’ for Rare Diseases But Ethical Guidelines Needed, Panel Says
May 30, 2018May 30, 2018
#ERDC2018 – When Treating Rare Disease Patients, Don’t Overlook Quality of Life, Panel Urges
May 29, 2018May 29, 2018
FDA Grants Fast-Track Designation for Krystal Biotech’s Gene Therapy Candidate KB103 to Treat DEB
May 25, 2018May 25, 2018

Positive Interim Results of Phase 1/2 Trial Support FCX-007 Gene Therapy for RDEB
May 23, 2018May 23, 2018
FCX-007, an experimental gene therapy developed by Fibrocell Science in collaboration with Precigen (a subsidiary ...
Trial Shows Abeona’s Gene Therapy EB-101 Helps Wounds Heal in RDEB Patients
May 21, 2018May 21, 2018
Diacerein Ointment Granted Rare Pediatric Disease Status for EB Treatment
May 17, 2018May 17, 2018

News

#ECRD2018 – EU Must Do More for Rare Disease Patients, Eurordis Leaders Say
May 17, 2018May 17, 2018
The European Union isn’t doing enough to protect the 30 million or so people ...
News

‘Rare Impact Awards’ Dinner Marks Orphan Drug Act and NORD at 35
May 17, 2018May 17, 2018
The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both ...
News

Rare Case of Genetic and Autoimmune Subtypes of EB Described in Report
May 16, 2018May 16, 2018
A rare case of autoimmune-driven epidermolysis bullosa (EB) was described in a patient who ...
News

Krystal Biotech Doses First Patient in Phase 1/2 Trial Testing Gene Therapy Candidate KB103 for DEB
May 14, 2018May 14, 2018
Krystal Biotech recently dosed the first patient in a Phase 1/2 clinical trial testing KB103, ...
News

Collagen Vesicles Released by Bone Marrow Stem Cells May Explain Their Therapeutic Potential, Study Shows
May 9, 2018May 9, 2018
Collagen type VII-filled vesicles released by bone marrow-derived mesenchymal stem cells may explain the ...
News

Epidermolysis Bullosa Can Contribute to Esophageal Stenosis, Case Study Finds
May 7, 2018May 7, 2018
Epidermolysis bullosa (EB) can affect the gastrointestinal tract, leading to the development of esophageal ...

January 23, 2018January 23, 2018

Factors Beyond COL7A1 Mutations May Contribute to Dystrophic EB Forms, Study Suggests

News

A study from Spain has raised the possibility that the development of dystrophic epidermolysis bullosa (DEB) could be attributable to additional factors besides mutations in the COL7A1 gene. Researchers reached this conclusion after studying three unrelated patients with different forms of ... Read more

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